The impact and treatment of developmental stuttering

This thesis is devoted to studying the impact of developmental stuttering in childhood and adulthood, and the outcomes of treatment in the preschool years

Psychometric evaluation of the Dutch translation of the Overall Assessment of the Speaker’s Experience of Stuttering for adults (OASES-A-D)

Abstract: The Overall Assessment of the Speaker’s Experience of Stuttering for adults (OASES-A; Yaruss & Quesal, 2006, 2010) is a patient-reported outcome measure that was designed to provide a comprehensive assessment of “the experience of the stuttering disorder from the perspective of individuals who stutter” (Yaruss & Quesal, 2006, p.90). This paper reports on the translation process and evaluates the psychometric performance of a Dutch version of the OASES-A. Translation of the OASES-A into Dutch followed a standard forward and backward translation process. The Dutch OASES-A (OASES-A-D) was then administered to 138 adults who stutter. A subset of 91 respondents also evaluated their speech on a 10-point Likert scale. For another subset of 45 respondents, a clinician-based stuttering severity rating on a 5-point Likert scale was available. Thirty-two of the respondents also completed the Dutch S-24 scale (Brutten & Vanryckeghem, 2003). The OASES-A-D showed acceptable item properties. No ceiling effects were observed. For 30 out of 100 items, most of which were in Section IV (Quality of Life), floor effects were observed. Cronbach’s alpha coefficients for all sections and subsections surpassed the 0.70 criterion of good internal consistency and reliability. Concurrent validity was moderate to high. Construct validity was confirmed by distinct scores on the OASES-A-D for groups with different levels of stuttering severity as rated by the speakers themselves or by clinicians. These results suggest that the OASES-A-D is a reliable and valid measure that can be used to assess the impact of stuttering on Dutch adults who stutter

Quality of Life in adults who stutter

Abstract: Although persistent developmental stuttering is known to affect daily living, just how great the impact is remains unclear. Furthermore, little is known about the underlying mechanisms which lead to a diminished quality of life (QoL). The primary objective of this study is to explore to what extent QoL is impaired in adults who stutter (AWS). In addition, this study aims to identify determinants of QoL in AWS by testing relationships between stuttering severity, coping, functioning and QoL and by testing for differences in variable scores between two AWS subgroups: receiving therapy versus not receiving therapy. A total of 91 AWS filled in several questionnaires to assess their stuttering severity, daily functioning, coping style and QoL. The QoL instruments used were the Health Utility Index 3 (HUI3) and the EuroQoL EQ-5D and EQ-VAS. The results indicated that moderate to severe stuttering has a negative impact on overall quality of life; HUI3 derived QoL values varied from .91 (for mild stuttering) to .73 (for severe stuttering). The domains of functioning that were predominantly affected were the individual’s speech, emotion, cognition and pain as measured by the HUI3 and daily activities and anxiety/depression as measured by the EQ-5D. AWS in the therapy group rated their stuttering as more severe and recorded more problems on the HUI3 speech domain than AWS in the non-therapy group. The EQ-VAS was the only instrument that showed a significant difference in overall QoL between groups. Finally, it was found that the relationship between stuttering severity and QoL was influenced by the individual’s coping style (emotion-oriented and task-oriented). These findings highlight the need for further research into stuttering in relation to QoL, and for a broader perspective on the diagnosis and treatment of stuttering, which would take into consideration quality of life and its determinants

Direct versus indirect treatment for preschool children who stutter: The RESTART randomized trial

Objective Stuttering is a common childhood disorder. There is limited high quality evidence regarding options for best treatment. The aim of the study was to compare the effectiveness of direct treatment with indirect treatment in preschool children who stutter. Methods In this multicenter randomized controlled trial with an 18 month follow-up, preschool children who stutter who were referred for treatment were randomized to direct treatment (Lidcombe Program; n = 99) or indirect treatment (RESTART-DCM treatment; n = 100). Main inclusion criteria were age 3-6 years, ≥ 3% syllables stuttered (%SS), and time since onset ≥ 6 months. The primary outcome was the percentage of non-stuttering children at 18 months. Secondary outcomes included stuttering frequency (%SS), stuttering severity ratings by the parents and therapist, severity rating by the child, health-related quality of life, emotional and behavioral problems, and speech attitude. Results Percentage of non-stuttering children for direct treatment was 76.5% (65/85) versus 71.4% (65/91) for indirect treatment (Odds Ratio (OR), 0.6; 95% CI, 0.1-2.4, p = .42). At 3 months, children treated by direct treatment showed a greater decline in %SS (significant interaction time x therapy: β = -1.89; t(282.82) = -2.807, p = .005). At 18 months, stuttering frequency was 1.2% (SD 2.1) for direct treatment and 1.5% (SD 2.1) for indirect treatment. Direct treatment had slightly better scores on most other secondary outcome measures, but no differences between treatment approaches were significant. Conclusions Direct treatment decreased stuttering more quickly during the first three months of treatment. At 18 months, however, clinical outcomes for direct and indirect treatment were comparable. These results imply that at 18 months post treatment onset, both treatments are roughly equal in treating developmental stuttering in ways that surpass expectations of natural recovery. Follow-up data are needed to confirm these findings in the longer term

Systematic review of available evidence on 11 high-priced inpatient orphan drugs

__Background__: Attention for Evidence Based Medicine (EBM) is growing, but evidence for orphan drugs is argued to be limited and inferior. This study systematically reviews the available evidence on clinical effectiveness, costeffectiveness and budget impact for orphan drugs. __Methods__: A systematic review was performed in PubMed, Embase, NHS EED and HTA databases for 11 inpatient orphan drugs listed on the Dutch policy rule on orphan drugs. For included studies, we determined the type of study and various study characteristics. __Results__: A total of 338 studies met all inclusion criteria. Almost all studies (96%) focused on clinical effectiveness of the drug. Of these studies, most studies were case studies (41%) or observational studies (39%). However, for all orphan diseases at least one experimental or quasi-experimental study was found, and a randomized clinical trial was available for 60% of the orphan drugs. Eight studies described the cost-effectiveness of an orphan drug; an equal number described an orphan drug’s budget impact. __Conclusions__: Despite the often heard claim that RCTs are not feasible for orphan drugs, we found that an RCT was available in 60% of o

Employment and associated impact on quality of life in people diagnosed with schizophrenia

A systematic review was conducted to assess the employment rate of people with schizophrenia. Additionally, information from the selected studies concerning factors associated with employment and health-related quality of life (HRQoL) was examined. Employment rates ranged from 4% to 50.4%. The studies differed considerably in design, patient settings, and methods of recruitment. The most frequently reported factors associated with employment were negative and cognitive symptoms, age of onset, and duration and course of the disease. Individual characteristics associated with unemployment were older age, lower education, and sex (female). Additionally, environmental factors, eg, the availability of welfare benefits and vocational support programs, seemed to play a role. Generally, being employed was positively associated with HRQoL. However, the causal direction of this association remained unclear, as studies on the bidirectional relationship between employment and HRQoL were lacking

Perspectives of Clinicians Involved in the RESTART-Study: Outcomes of a Focus Group

PURPOSE: The purpose of this study was to explore the attitudes and beliefs of speech-language pathologists (SLPs) with regard to the Lidcombe Program and Demands and Capacities-based treatment and to examine how these attitudes and beliefs might have changed as a result of participating in the RESTART-study. METHOD: A focus group meeting with 13 SLPs was organized. The discussion was structured using questions on therapy preference, attitudes about and explicit comparison of both treatments and treatment manuals, and learnings of trial participation. RESULTS: Four main themes were identified. First, a change in attitude toward treatment choice was observed. Second, this change was related to a change in beliefs about the potential of both treatments. Third, aspects of the treatments regarded as success factors were considered. Last, learning outcomes and increased professionalism as a result of participating in the RESTART-trial were discussed. CONCLUSIONS: This study showed how attitudes and beliefs of SLPs with regard to the Lidcombe Program and Demands and Capacities-based treatment evolved during a randomized trial. This work increases our understanding of the role of attitudes and beliefs in the uptake and utilization of therapies and demonstrates the importance of collecting qualitative data. Results and recommendations should prove of value in implementing the RESTART-trial results and in training SLPs